RiboX Therapeutics' RXRG001 Receives FDA IND Clearance as First Circular RNA Therapy for Radiation-Induced Xerostomia
核心洞察
RiboX Therapeutics (搜索) received FDA clearance for its IND application for RXRG001, a circular RNA therapy, to treat radiation-induced xerostomia (搜索) and hyposalivation (搜索).
The SPRINX-1 study, a Phase I/IIa trial, will assess the safety and efficacy of RXRG001 in patients suffering from dry mouth and low saliva secretion due to radiation.
RXRG001 leverages circular RNA technology to overexpress human aquaporin 1 (搜索) (hAQP1 (搜索)), aiming to restore water permeability and alleviate dry mouth symptoms.
RiboX Therapeutics (搜索) Ltd. has announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for RXRG001, marking it as the first circular RNA therapy to enter clinical development. The Phase I/IIa study (SPRINX-1) is set to evaluate the safety and efficacy of RXRG001 in patients with radiation-induced xerostomia (搜索) (RIX) and hyposalivation (搜索).
Addressing an Unmet Need in Head and Neck Cancer Treatment
Xerostomia and hyposalivation (搜索) are common and debilitating side effects of radiation therapy in head and neck cancer (搜索) (HNC) patients. With the incidence of HNC increasing globally, radiation therapy, while crucial for survival, often damages salivary glands, leading to decreased saliva secretion and impaired quality of life. Current treatments are often ineffective, temporary, and costly, highlighting a significant unmet medical need.
RXRG001: A Novel Circular RNA Therapeutic Approach
RXRG001 is a proprietary product utilizing circular RNA technology, which has emerged as a next-generation mRNA therapy due to its high protein expression efficiency, low immunogenicity, and manufacturing stability. The therapy consists of circular RNA coding for human aquaporin 1 (搜索) (hAQP1 (搜索)), a water channel protein, encapsulated in lipid nanoparticles (LNPs). By overexpressing hAQP1, RXRG001 aims to restore water permeability and increase saliva production, thereby alleviating dry mouth symptoms.
Promising Preclinical Results
Non-clinical studies have demonstrated a favorable risk and benefit profile for RXRG001. Notably, a single administration of RXRG001 in animal models resulted in a significant and sustained increase in salivary flow lasting approximately four weeks.
Expert Perspectives
"RIX is a devastating life-long health issue for patients with HNC. Although radiation therapy improves patients’ survival, its damage to the salivary glands leads to an impaired quality of life of many cancer survivors," said Dr. Yizhen Xu, Chief Medical Officer of RiboX. "RXRG001 may offer a potential effective and sustained therapeutic option for RIX patients. We are excited to conduct clinical trials to further evaluate its efficacy and safety in patients."
Dr. Weiyi Zhang, Chief Executive Officer of RiboX, added, "The FDA’s clearance of the IND application for RXRG001 is an acknowledgment of our innovative capabilities, robust technology, and competitiveness in the field of circular RNA therapeutics. RiboX will advance the clinical development of RXRG001, continue leveraging the unique advantages of circular RNA technology in therapeutic applications, and introduce more pioneering treatments to patients around the world."